EP2061443A4 - Polykonjugate zur in-vivo-verabreichung von polynukleotiden - Google Patents

Polykonjugate zur in-vivo-verabreichung von polynukleotiden

Info

Publication number
EP2061443A4
EP2061443A4 EP07841047.9A EP07841047A EP2061443A4 EP 2061443 A4 EP2061443 A4 EP 2061443A4 EP 07841047 A EP07841047 A EP 07841047A EP 2061443 A4 EP2061443 A4 EP 2061443A4
Authority
EP
European Patent Office
Prior art keywords
polyconjugates
polynucleotides
vivo delivery
vivo
delivery
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP07841047.9A
Other languages
English (en)
French (fr)
Other versions
EP2061443A2 (de
Inventor
David B Rozema
Darren H Wakefield
Jon A Wolff
So Wong
Jason Klein
David L Lewis
James E Hagstrom
Andrei Blokhin
Vladimir Trubetskoy
Hans Herweijer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arrowhead Pharmaceuticals Inc
Original Assignee
Arrowhead Research Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arrowhead Research Corp filed Critical Arrowhead Research Corp
Publication of EP2061443A2 publication Critical patent/EP2061443A2/de
Publication of EP2061443A4 publication Critical patent/EP2061443A4/de
Withdrawn legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/74—Synthetic polymeric materials
    • A61K31/785—Polymers containing nitrogen
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/58—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A01K2217/058—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to expression of inhibitory nucleic acid, e.g. siRNA, antisense
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0362—Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
EP07841047.9A 2006-08-18 2007-08-17 Polykonjugate zur in-vivo-verabreichung von polynukleotiden Withdrawn EP2061443A4 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US82283306P 2006-08-18 2006-08-18
US91586807P 2007-05-03 2007-05-03
PCT/US2007/076202 WO2008022309A2 (en) 2006-08-18 2007-08-17 Polyconjugates for in vivo delivery of polynucleotides
US11/840,468 US8137695B2 (en) 2006-08-18 2007-08-17 Polyconjugates for in vivo delivery of polynucleotides

Publications (2)

Publication Number Publication Date
EP2061443A2 EP2061443A2 (de) 2009-05-27
EP2061443A4 true EP2061443A4 (de) 2013-07-24

Family

ID=39083168

Family Applications (1)

Application Number Title Priority Date Filing Date
EP07841047.9A Withdrawn EP2061443A4 (de) 2006-08-18 2007-08-17 Polykonjugate zur in-vivo-verabreichung von polynukleotiden

Country Status (13)

Country Link
US (4) US8137695B2 (de)
EP (1) EP2061443A4 (de)
JP (2) JP5274461B2 (de)
KR (1) KR101133799B1 (de)
CN (2) CN102614528B (de)
AU (1) AU2007285782B2 (de)
BR (1) BRPI0715375A2 (de)
CA (1) CA2660842C (de)
IL (1) IL196508A (de)
MX (1) MX2009001207A (de)
NO (1) NO20090422L (de)
WO (1) WO2008022309A2 (de)
ZA (1) ZA200900491B (de)

Families Citing this family (212)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
WO2006042112A2 (en) 2004-10-05 2006-04-20 California Institute Of Technology Aptamer regulated nucleic acids and uses thereof
US8658211B2 (en) * 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
WO2008058291A2 (en) * 2006-11-09 2008-05-15 California Institute Of Technology Modular aptamer-regulated ribozymes
AR066984A1 (es) 2007-06-15 2009-09-23 Novartis Ag Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia)
US20090082217A1 (en) * 2007-07-16 2009-03-26 California Institute Of Technology Selection of nucleic acid-based sensor domains within nucleic acid switch platform
US20120165387A1 (en) 2007-08-28 2012-06-28 Smolke Christina D General composition framework for ligand-controlled RNA regulatory systems
US8367815B2 (en) * 2007-08-28 2013-02-05 California Institute Of Technology Modular polynucleotides for ligand-controlled regulatory systems
US8865667B2 (en) 2007-09-12 2014-10-21 California Institute Of Technology Higher-order cellular information processing devices
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
WO2009102782A2 (en) * 2008-02-11 2009-08-20 Bico Scientific Corporation Increasing efficiency of nucleic acid delivery in vivo using targeting conjugates
JP2009203173A (ja) * 2008-02-26 2009-09-10 Hokkaido Univ ｓｉＲＮＡ−ポリカチオン複合体を含有するイオントフォレーシス用組成物
WO2009126933A2 (en) * 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US9339558B2 (en) 2008-05-13 2016-05-17 University Of Washington Micellic assemblies
CA2724105C (en) 2008-05-13 2017-09-05 University Of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
US9006193B2 (en) 2008-05-13 2015-04-14 University Of Washington Polymeric carrier
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
US7842725B2 (en) 2008-07-24 2010-11-30 Ecolab USA, Inc. Foaming alcohol compositions with selected dimethicone surfactants
WO2010021770A1 (en) * 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
US8796443B2 (en) 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
CA2742955A1 (en) 2008-11-06 2010-05-14 University Of Washington Bispecific intracellular delivery vehicles
KR20110095292A (ko) 2008-11-06 2011-08-24 유니버시티 오브 워싱톤 다중블록 공중합체
CA2742846A1 (en) * 2008-11-17 2010-05-20 Enzon Pharmaceuticals, Inc. Releasable fusogenic lipids for nucleic acids delivery systems
EP2373715A4 (de) 2008-12-08 2014-12-03 Univ Washington Omega-funktionalisierte polymere, übergangsfunktionalisierte blockcopolymere, polymerbiokonjugate und radikalische kettenverlängerungspolymerisaktion
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
GB0901699D0 (en) * 2009-02-02 2009-03-11 Ntnu Technology Transfer As gas seperation membrane
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US8329882B2 (en) 2009-02-18 2012-12-11 California Institute Of Technology Genetic control of mammalian cells with synthetic RNA regulatory systems
EP2403863B1 (de) 2009-03-02 2013-08-28 Alnylam Pharmaceuticals Inc. Chemische modifikationen von nukleinsäure
US9145555B2 (en) 2009-04-02 2015-09-29 California Institute Of Technology Integrated—ligand-responsive microRNAs
EP2451479B1 (de) 2009-07-06 2015-04-01 F.Hoffmann-La Roche Ag Bispezifische digoxigenin-bindungs-antikörper
WO2011007894A1 (ja) * 2009-07-15 2011-01-20 住友化学株式会社 ポリビニルエーテル樹脂
EP3252068A3 (de) 2009-10-12 2018-03-14 Larry J. Smith Verfahren und zusammensetzungen zur modulation der genexpression unter verwendung in-vivo- oder in-vitro-verabreichten arzneistoffen auf oligonukleotidbasis
WO2011053614A1 (en) * 2009-10-30 2011-05-05 Merck Sharp & Dohme Corp. Crystalline cdm-nag and methods for producing same
US20130017167A1 (en) * 2009-11-13 2013-01-17 University Of Washington Through Its Center For Commercialization Hydrophobic block conjugated therapeutic agents
US9415113B2 (en) 2009-11-18 2016-08-16 University Of Washington Targeting monomers and polymers having targeting blocks
US8765432B2 (en) 2009-12-18 2014-07-01 Oligasis, Llc Targeted drug phosphorylcholine polymer conjugates
US8470891B2 (en) * 2009-12-23 2013-06-25 International Business Machines Corporation Biodegradable block polymers for drug delivery, and methods related thereto
US20110151566A1 (en) * 2009-12-23 2011-06-23 James Hedrick Biodegradable polymers, complexes thereof for gene therapeutics and drug delivery, and methods related thereto
US8361495B2 (en) * 2009-12-23 2013-01-29 International Business Machines Corporation Antimicrobial polymers and methods of manufacture thereof
EP3023495B1 (de) 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Zusammensetzung für die gezielte freisetzung von sirna
EP2550002B1 (de) 2010-03-24 2019-05-08 Phio Pharmaceuticals Corp. Rna-interferenz bei dermalen und fibrotischen erkrankungen
WO2011119871A1 (en) 2010-03-24 2011-09-29 Rxi Phrmaceuticals Corporation Rna interference in ocular indications
EP2553019A1 (de) 2010-03-26 2013-02-06 Mersana Therapeutics, Inc. Modifizierte polymere zur freisetzung von polynukleotiden, verfahren zu ihrer herstellung und verfahren zu ihrer verwendung
KR102416359B1 (ko) 2010-04-15 2022-07-01 코디악 사이언시스 인코포레이티드 고분자량 쌍성이온-함유 중합체
WO2011133868A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Conformationally restricted dinucleotide monomers and oligonucleotides
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2011161075A1 (en) 2010-06-22 2011-12-29 Dna Therapeutics Optimized in vivo delivery system with endosomolytic agents for nucleic acid conjugates
JP6043285B2 (ja) 2010-08-02 2016-12-14 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低分子干渉核酸（ｓｉＮＡ）を用いたカテニン（カドヘリン結合型タンパク質）β１（ＣＴＮＮＢ１）遺伝子発現のＲＮＡ干渉媒介性阻害
CA2807440A1 (en) 2010-08-04 2012-02-09 Cizzle Biotechnology Limited Methods and compounds for the diagnosis and treatment of cancer
HUE044815T2 (hu) 2010-08-17 2019-11-28 Sirna Therapeutics Inc Hepatitisz B vírus (HBV) génexpressziójának RNS-interferencia közvetített gátlása, rövid interferáló nukleinsav (SINS) alkalmazásával
WO2012027206A1 (en) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
US9233997B2 (en) 2010-08-26 2016-01-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of prolyl hydroxylase domain 2 (PHD2) gene expression using short interfering nucleic acid (siNA)
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
JP2014504295A (ja) 2010-12-17 2014-02-20 アローヘッド リサーチ コーポレイション ｓｉＲＮＡ用ガラクトースクラスター−薬物動態調節剤標的指向部分
CN103491982B (zh) * 2010-12-29 2017-09-12 箭头研究公司 具有酶敏感性连接的多核苷酸体内递送偶联物
EP2658981B1 (de) 2010-12-29 2016-09-28 F.Hoffmann-La Roche Ag Kleinmolekülige konjugate zur intrazellulären freisetzung von nukleinsäuren
EP2527440A1 (de) 2011-05-27 2012-11-28 Institut Curie Krebsbehandlung durch Kombination von Doppelstrangbruch nachahmenden DNA-Molekülen mit Hyperthermie
WO2012173784A1 (en) 2011-06-17 2012-12-20 Arrowhead Madison Inc. Disubstituted maleic anhydrides with altered kinetics of ring closure
BR112014004585A2 (pt) 2011-08-26 2017-06-13 Arrowhead Res Corp polímeros de poli(éster vinílico) para liberação de ácido nucleico in vivo
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
JP6129844B2 (ja) 2011-09-14 2017-05-17 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物
WO2013062982A1 (en) * 2011-10-27 2013-05-02 Merck Sharp & Dohme Corp. Poly(lysine) homopolymers for the delivery of oligonucleotides
WO2013158141A1 (en) * 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP3919620A1 (de) 2012-05-02 2021-12-08 Sirna Therapeutics, Inc. Zusammensetzungen mit kurzer interferierender nukleinsäure (sina)
CN104662000B (zh) 2012-05-15 2018-08-17 索伦托医疗有限公司 药物偶联物及其偶联方法和用途
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
DE102012022596B4 (de) * 2012-11-15 2017-05-04 Friedrich-Schiller-Universität Jena Neue zellspezifisch wirksame Nukleotid-Moleküle und Applikationskit zu deren Anwendung
BR112015012840A2 (pt) 2012-12-06 2017-07-11 Merck Sharp & Dohme composto, composição, e, uso de um composto ou um sal, solvato, éster ou pró-droga farmaceuticamente aceitável do mesmo
EP2961843A2 (de) 2013-02-28 2016-01-06 Arrowhead Research Corporation Organische zusammensetzungen zur behandlung von epas1-bedingten erkrankungen
EP3633039A1 (de) 2013-05-01 2020-04-08 Ionis Pharmaceuticals, Inc. Zusammensetzungen und verfahren
CA2919828C (en) 2013-07-30 2022-07-19 Phaserx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
EA201690051A1 (ru) 2013-08-07 2016-06-30 Эрроухэд Рисерч Корпорейшн Поликонъюгаты для доставки триггеров рнк интерференции в клетки опухоли in vivo
WO2015069586A2 (en) * 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Dual molecular delivery of oligonucleotides and peptide containing conjugates
KR20160110370A (ko) 2013-12-04 2016-09-21 알엑스아이 파마슈티칼스 코포레이션 화학적으로 변형된 올리고뉴클레오티드를 사용한 상처 치유의 치료 방법
US9926556B2 (en) 2014-04-28 2018-03-27 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
CA2947619A1 (en) 2014-05-01 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treatment of disorders in the front of the eye utilizing nucleic acid molecules
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
PE20170010A1 (es) 2014-05-01 2017-03-04 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
CN106255755B (zh) 2014-05-01 2020-07-24 Ionis制药公司 用于调节血管生成素样蛋白3表达的组合物和方法
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
CN107073294A (zh) 2014-09-05 2017-08-18 阿克赛医药公司 使用靶向tyr或mmp1的核酸治疗老化和皮肤病症的方法
WO2016069694A2 (en) 2014-10-30 2016-05-06 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
CN107249678B (zh) * 2014-12-19 2021-02-19 凯敏工业公司 使用离子电渗法进行生物活性分子的眼内递送
EP3034539A1 (de) * 2014-12-19 2016-06-22 Ethris GmbH Zusammensetzungen zur Einführung von Nukleinsäure in Zellen
CN107530436B (zh) 2015-01-21 2022-03-29 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
CA2979703A1 (en) 2015-03-17 2016-09-22 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor xii
CN107635550B (zh) * 2015-03-17 2021-12-24 箭头药业股份有限公司 改良的含二硫化物的炔烃交联剂
AU2016257150B2 (en) 2015-05-06 2022-03-31 Benitec IP Holdings Inc. Reagents for treatment of hepatitis B virus (HBV) infection and use thereof
CN108064156B (zh) 2015-05-29 2022-02-01 箭头药业股份有限公司 抑制Hif2α基因表达的组合物及方法
MX2017014638A (es) 2015-05-29 2018-01-23 Arrowhead Pharmaceuticals Inc Agentes enlazantes tetrapeptidicos escindibles biologicamente.
CA2991598A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
EP4092119A3 (de) 2015-07-10 2023-03-22 Ionis Pharmaceuticals, Inc. Modulatoren der diacyglycerol-acyltransferase (2 dgat2)
US11053495B2 (en) 2015-07-17 2021-07-06 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
LT3325623T (lt) 2015-07-23 2019-10-25 Inst Curie Dbait molekulės ir parp inhibitorių derinio panaudojimas vėžiui gydyti
CN108271387B (zh) 2015-08-07 2023-06-27 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
AR106135A1 (es) 2015-09-24 2017-12-13 Ionis Pharmaceuticals Inc Moduladores de la expresión del sarcoma de la rata de kirsten (kras)
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
EP4119569A1 (de) 2015-11-06 2023-01-18 Ionis Pharmaceuticals, Inc. Konjugierte antisense-verbindungen zur verwendung in der therapie
JP2018531605A (ja) 2015-11-06 2018-11-01 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． アポリポタンパク質（ａ）発現の調節
US20200123499A1 (en) 2015-12-02 2020-04-23 Massachusetts Institute Of Technology Method for efficient generation of neurons from non-neuronal cells
BR112018011450A2 (pt) 2015-12-07 2018-11-27 Genzyme Corp métodos e composições para o tratamento de um transtorno associado à serpinc1
IL302398A (en) 2016-03-03 2023-06-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
TWI783434B (zh) 2016-03-07 2022-11-11 美商愛羅海德製藥公司 治療性化合物之標靶性配體
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2017186882A1 (en) 2016-04-29 2017-11-02 Onxeo A method of predicting a response to an anti-tumor treatment by means of signal interfering dna molecules
CN109526222B (zh) 2016-06-06 2022-04-29 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
CN109844115B (zh) 2016-07-15 2023-06-30 Ionis 制药公司 用于调节smn2的化合物和方法
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
UY37376A (es) 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
TW202320855A (zh) 2016-09-02 2023-06-01 美商愛羅海德製藥公司 標靶性配體
HUE059718T2 (hu) 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
EP3522898A4 (de) 2016-10-06 2020-05-27 Ionis Pharmaceuticals, Inc. Verfahren zum konjugieren von oligomeren verbindungen
EP3562510A4 (de) 2016-12-30 2021-01-06 Genevant Sciences GmbH Verzweigte peg-moleküle sowie zugehörige zusammensetzungen und verfahren
CA3045045A1 (en) 2017-01-10 2018-07-19 Zhen Li Alpha-1 antitrypsin (aat) rnai agents, compositions including aat rnai agents, and methods of use
WO2018162439A1 (en) 2017-03-08 2018-09-13 Onxeo New predictive biomarker for the sensitivity to a treatment of cancer with a dbait molecule
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
BR112019023650A2 (pt) 2017-07-06 2020-06-02 Arrowhead Pharmaceuticals, Inc. Agentes rnai para inibir a expressão de alfa-enac e métodos de uso
JP7282379B2 (ja) 2017-08-22 2023-05-29 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
SG11201912178VA (en) 2017-09-11 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
US10995335B2 (en) 2017-09-14 2021-05-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
EP3461488A1 (de) 2017-09-27 2019-04-03 Onxeo Kombination aus einem dbait-molekül und einem hdac-inhibitor zur behandlung von krebs
CN111212909A (zh) 2017-10-17 2020-05-29 箭头药业股份有限公司 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物
KR20200098521A (ko) 2017-11-13 2020-08-20 사일런스 테라퓨틱스 게엠베하 포스포로다이티오에이트 링키지를 포함하는 표적 유전자의 발현을 저해하기 위한 핵산
CN111902148A (zh) 2017-12-06 2020-11-06 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
EA202091513A1 (ru) 2017-12-19 2020-09-09 Янссен Сайенсиз Айрлэнд Анлимитед Компани Вакцины против вируса гепатита b (hbv) и их применение
MA51645A (fr) 2018-01-15 2020-11-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression de dnm2
US11332733B2 (en) 2018-02-12 2022-05-17 lonis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP2021515580A (ja) 2018-03-13 2021-06-24 オンクセオＯｎｘｅｏ がんの治療における獲得耐性に対抗するｄｂａｉｔ分子
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
BR112020020957B1 (pt) 2018-05-09 2022-05-10 Ionis Pharmaceuticals, Inc Compostos oligoméricos, população e composição farmacêutica dos mesmos e seus usos
WO2020033748A1 (en) 2018-08-08 2020-02-13 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
TW202028465A (zh) 2018-09-28 2020-08-01 美商阿尼拉製藥公司 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其治療或預防ＴＴＲ相關眼部疾病之使用方法
KR20210109564A (ko) 2018-12-21 2021-09-06 옹쎄오 신규의 컨쥬게이티드 핵산 분자 및 이의 용도
AU2020219355A1 (en) 2019-02-07 2021-09-30 Arrowhead Pharmaceuticals, Inc. RNAi agents for Hepatitis B virus infection
EP3941528A1 (de) 2019-03-21 2022-01-26 Codiak BioSciences, Inc. Extrazelluläre vesikelkonjugate und verwendungen davon
WO2020188015A1 (en) 2019-03-21 2020-09-24 Onxeo A dbait molecule in combination with kinase inhibitor for the treatment of cancer
CN114080232A (zh) 2019-03-21 2022-02-22 科迪亚克生物科学公司 用于疫苗递送的细胞外囊泡
BR112021018739A2 (pt) 2019-03-29 2022-05-03 Dicerna Pharmaceuticals Inc Composições e métodos para o tratamento de doenças ou distúrbios associados a kras
KR20220009389A (ko) 2019-04-17 2022-01-24 코디악 바이오사이언시즈, 인크. 엑소좀 및 aav 의 조성물
CA3132170A1 (en) 2019-04-18 2020-10-22 Michael BIERMER Combination therapy for treating hepatitis b virus infection
WO2020214974A1 (en) 2019-04-18 2020-10-22 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
BR112021021686A2 (pt) 2019-05-03 2022-03-22 Dicerna Pharmaceuticals Inc Moléculas inibidoras de ácido nucleico de fita dupla com fitas senso curtas
CA3138915A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
CN114340664A (zh) 2019-06-18 2022-04-12 爱尔兰詹森科学公司 乙型肝炎病毒(HBV)疫苗和靶向HBV的RNAi的组合
US20220251200A1 (en) 2019-07-03 2022-08-11 Codiak Biosciences, Inc. Extracellular vesicles targeting t cells and uses thereof
WO2021030768A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with stat3-antisense oligonucleotides
CN114729356A (zh) 2019-08-14 2022-07-08 科迪亚克生物科学公司 靶向stat6的细胞外囊泡-aso构建体
MX2022001769A (es) 2019-08-14 2022-06-09 Codiak Biosciences Inc Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta.
US20230132093A1 (en) 2019-08-14 2023-04-27 Codiak Biosciences, Inc. Extracellular vesicle-nlrp3 antagonist
CN114728078A (zh) 2019-08-14 2022-07-08 科迪亚克生物科学公司 与分子连接的细胞外囊泡及其用途
WO2021030781A1 (en) 2019-08-14 2021-02-18 Codiak Biosciences, Inc. Extracellular vesicles with antisense oligonucleotides targeting kras
EP4041312A4 (de) 2019-10-10 2023-12-20 Kodiak Sciences Inc. Verfahren zur behandlung einer augenstörung
JP2022552249A (ja) 2019-10-14 2022-12-15 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
KR20220110749A (ko) 2019-11-06 2022-08-09 알닐람 파마슈티칼스 인코포레이티드 간외 전달
WO2021092145A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna composition and methods of use thereof for treating or preventing ttr-associated ocular diseases
US20230002766A1 (en) 2019-11-08 2023-01-05 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
EP4085136A1 (de) 2019-12-31 2022-11-09 Phio Pharmaceuticals Corp. Chemisch modifizierte oligonukleotide mit verbesserter systemischer abgabe
AU2021207504A1 (en) 2020-01-15 2022-08-04 Dicerna Pharmaceuticals, Inc. 4'-O-methylene phosphonate nucleic acids and analogues thereof
KR20220148230A (ko) 2020-02-28 2022-11-04 아이오니스 파마수티컬즈, 인코포레이티드 Smn2를 조절하기 위한 화합물 및 방법
TW202146011A (zh) 2020-03-05 2021-12-16 美商詹森藥物公司 治療b型肝炎病毒感染之組合療法
EP4121063A1 (de) 2020-03-19 2023-01-25 Avidity Biosciences, Inc. Zusammensetzungen und verfahren zur behandlung von fazioskapulohumeraler muskeldystrophie
CA3172591A1 (en) 2020-03-24 2021-09-30 Douglas Anthony KERR Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
KR20220158011A (ko) 2020-03-26 2022-11-29 애로우헤드 파마슈티컬스 인코포레이티드 PNPLA3의 발현을 억제하기 위한 RNAi 작용제, 이의 약제학적 조성물, 및 사용 방법
TW202210633A (zh) 2020-06-05 2022-03-16 法商昂席歐公司 用於治療癌症之dbait分子與kras抑制劑的組合
TW202214857A (zh) 2020-06-19 2022-04-16 法商昂席歐公司 新型結合核酸分子及其用途
US20210403908A1 (en) 2020-06-22 2021-12-30 Janssen Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis d virus infection
CN111760556B (zh) * 2020-07-09 2023-06-30 江苏尤里卡生物科技有限公司 一种尿激酶吸附剂及其制备方法和应用
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
EP4192505A1 (de) 2020-08-04 2023-06-14 Dicerna Pharmaceuticals, Inc. Systemische verabreichung von oligonukleotiden
UY39420A (es) 2020-09-11 2022-03-31 Arrowhead Pharmaceuticals Inc Conjugados lipídicos para el transporte de agentes terapéuticos
UY39417A (es) 2020-09-11 2022-03-31 Arrowhead Pharmaceuticals Inc Agentes de arni para inhibir la expresión de dux4, composiciones de dichos agentes, y métodos de uso
TW202218687A (zh) 2020-09-11 2022-05-16 美商愛羅海德製藥公司 骨骼肌遞送載體及其使用方法
EP4216993A2 (de) 2020-09-23 2023-08-02 Codiak BioSciences, Inc. Verfahren zur herstellung von extrazellulären vesikeln
WO2022066883A1 (en) 2020-09-23 2022-03-31 Codiak Biosciences, Inc. Extracellular vesicles comprising kras antigens and uses thereof
WO2022076596A1 (en) 2020-10-06 2022-04-14 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
JP2023549563A (ja) 2020-11-18 2023-11-27 アイオーニス ファーマシューティカルズ， インコーポレーテッド アンジオテンシノーゲン発現を調節するための化合物及び方法
TW202245809A (zh) 2020-12-18 2022-12-01 美商詹森藥物公司 用於治療b型肝炎病毒感染之組合療法
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
WO2022147214A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2022152869A1 (en) 2021-01-15 2022-07-21 Janssen Sciences Ireland Unlimited Company Use of oligonucleotides for individuals with hepatic impairment
JP2024506711A (ja) 2021-02-17 2024-02-14 ロンザ セールス アーゲー 最適化リンカー及びアンカー部分を介して生物学的に活性な分子に連結した細胞外ベシクル
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
CN113292616B (zh) * 2021-05-20 2023-05-23 内蒙古大学 一种单糖配体功能化的阳离子脂质类化合物及其制备方法与应用
US11629349B2 (en) 2021-06-21 2023-04-18 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
CA3224600A1 (en) 2021-07-09 2023-01-12 Glaxosmithkline Intellectual Property (No.3) Limited Use of oligonucleotides for individuals with renal impairment
KR20240036041A (ko) 2021-07-21 2024-03-19 알닐람 파마슈티칼스 인코포레이티드 대사 장애-연관 표적 유전자 iRNA 조성물 및 이의 사용 방법
WO2023015264A1 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
WO2023015265A2 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023064530A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
WO2023111203A1 (en) 2021-12-16 2023-06-22 Onxeo New conjugated nucleic acid molecules and their uses
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
WO2023245060A2 (en) 2022-06-15 2023-12-21 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
KR20240047851A (ko) 2022-10-05 2024-04-12 성균관대학교산학협력단 종양 환경 감응형 절단성 폴리에틸렌글리콜이 결합된 암 치료용 항체 및 이의 제조방법

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040156909A1 (en) * 2003-02-07 2004-08-12 Rozema David B. Polyvinylethers for delivery of polynucleotides to mammalian cells
US20040162260A1 (en) * 2003-02-18 2004-08-19 Rozema David B. Reversible attachment of a membrane active polymer to a polynucleotide
US20040198687A1 (en) * 2003-04-04 2004-10-07 Rozema David B. Endosomolytic polymers

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4587044A (en) * 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5166320A (en) * 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US4904582A (en) * 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5138045A (en) * 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
EP1236473A3 (de) * 1992-04-03 2003-01-15 The Regents Of The University Of California Selbst zusammenbaubares system zur verabreichung von polynukleotiden
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
JP2690276B2 (ja) * 1995-01-10 1997-12-10 科学技術振興事業団 静電結合型高分子ミセル薬物担体とその薬剤
AU1039397A (en) * 1995-11-22 1997-06-27 Johns Hopkins University, The Ligands to enhance cellular uptake of biomolecules
US20020165183A1 (en) * 1999-11-29 2002-11-07 Hans Herweijer Methods for genetic immunization
US5994316A (en) * 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US6020457A (en) * 1996-09-30 2000-02-01 Dendritech Inc. Disulfide-containing dendritic polymers
GB9623051D0 (en) * 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems
DE69834038D1 (de) * 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
US5877309A (en) * 1997-08-13 1999-03-02 Isis Pharmaceuticals, Inc. Antisense oligonucleotides against JNK
US6300319B1 (en) * 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US8038984B2 (en) * 1998-06-20 2011-10-18 Washington University Membrane-permeant peptide complexes for treatment of sepsis
US20050112470A1 (en) * 1998-06-26 2005-05-26 Johnson Controls Technology Company Alloy for battery grids
TWI242000B (en) 1998-12-10 2005-10-21 Univ Southern California Reversible aqueous pH sensitive lipidizing reagents, compositions and methods of use
US6773920B1 (en) * 1999-03-31 2004-08-10 Invitrogen Corporation Delivery of functional protein sequences by translocating polypeptides
US7019113B2 (en) * 1999-06-07 2006-03-28 Mirus Bio Corporation Reversible modification of membrane interaction
DK1102785T3 (da) * 1999-06-07 2013-05-13 Arrowhead Res Corp Sammensætninger til lægemiddeltilførsel ved anvendelse af pH-følsomme molekyler
US8211468B2 (en) * 1999-06-07 2012-07-03 Arrowhead Madison Inc. Endosomolytic polymers
US7442764B2 (en) * 1999-06-07 2008-10-28 Mirns Bio Corporation Reversible modification of amine-containing compounds
US20040072785A1 (en) * 1999-11-23 2004-04-15 Wolff Jon A. Intravascular delivery of non-viral nucleic acid
US7098030B2 (en) * 1999-12-31 2006-08-29 Mirus Bio Corporation Polyampholytes for delivering polyions to a cell
US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) * 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US20030072794A1 (en) * 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
US7807780B2 (en) * 2000-07-21 2010-10-05 Revance Therapeutics, Inc. Multi-component biological transport systems
US7427394B2 (en) * 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US6998115B2 (en) * 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
IL161733A0 (en) * 2001-11-02 2005-11-20 Insert Therapeutics Inc Methods and compositions for therapeutic use of rna interference
US7176303B2 (en) * 2003-11-06 2007-02-13 Isis Pharmaceuticals, Inc. Modulation of STAT5 expression
US7220400B2 (en) * 2002-05-24 2007-05-22 Mirus Bio Corporation Compositions for delivering nucleic acids to cells
WO2004013307A2 (en) * 2002-08-05 2004-02-12 Mirus Corporation Compounds for targeting hepatocytes
KR20050084607A (ko) * 2002-09-28 2005-08-26 매사추세츠 인스티튜트 오브 테크놀로지 인플루엔자 치료제
EP1585756B1 (de) * 2002-11-26 2010-04-21 University of Massachusetts Verabreichung von sirnas
WO2004087931A1 (en) * 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20060008907A1 (en) * 2004-06-09 2006-01-12 The Curators Of The University Of Missouri Control of gene expression via light activated RNA interference
WO2006053430A1 (en) * 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
US7923206B2 (en) * 2004-11-22 2011-04-12 Dharmacon, Inc. Method of determining a cellular response to a biological agent
EP1907444B1 (de) * 2005-04-01 2009-08-19 Intezyne Technologies Incorporated Polymermicellen für die arzneistoffzufuhr
US9139850B2 (en) * 2005-05-19 2015-09-22 L'oreal Vectorization of dsRNA by cationic particles and topical use
CA2637931A1 (en) * 2006-01-23 2007-07-26 Abbott Laboratories Chemically modified polycation polymer for sirna delivery

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040156909A1 (en) * 2003-02-07 2004-08-12 Rozema David B. Polyvinylethers for delivery of polynucleotides to mammalian cells
US20040162260A1 (en) * 2003-02-18 2004-08-19 Rozema David B. Reversible attachment of a membrane active polymer to a polynucleotide
US20040198687A1 (en) * 2003-04-04 2004-10-07 Rozema David B. Endosomolytic polymers

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
OMOLOLA ENIOLA A ET AL: "In vitro characterization of leukocyte mimetic for targeting therapeutics to the endothelium using two receptors", BIOMATERIALS, ELSEVIER SCIENCE PUBLISHERS BV., BARKING, GB, vol. 26, no. 34, 1 December 2005 (2005-12-01), pages 7136 - 7144, XP027767685, ISSN: 0142-9612, [retrieved on 20051201] *

Also Published As

Publication number Publication date
AU2007285782B2 (en) 2010-06-24
US20080281044A1 (en) 2008-11-13
JP5735573B2 (ja) 2015-06-17
NO20090422L (no) 2009-02-26
JP2013215193A (ja) 2013-10-24
JP5274461B2 (ja) 2013-08-28
US7985406B2 (en) 2011-07-26
WO2008022309A2 (en) 2008-02-21
US20080152661A1 (en) 2008-06-26
CA2660842C (en) 2012-03-13
EP2061443A2 (de) 2009-05-27
BRPI0715375A2 (pt) 2013-06-18
IL196508A (en) 2016-04-21
US20090023890A1 (en) 2009-01-22
AU2007285782A1 (en) 2008-02-21
US20080269450A1 (en) 2008-10-30
ZA200900491B (en) 2009-12-30
CN101500548A (zh) 2009-08-05
WO2008022309A3 (en) 2008-11-20
KR101133799B1 (ko) 2012-04-24
KR20090042918A (ko) 2009-05-04
US8137695B2 (en) 2012-03-20
CA2660842A1 (en) 2008-02-21
IL196508A0 (en) 2009-11-18
JP2010521133A (ja) 2010-06-24
CN102614528A (zh) 2012-08-01
MX2009001207A (es) 2009-02-11
CN102614528B (zh) 2014-02-26

Similar Documents

Publication Publication Date Title
IL196508A0 (en) Polyconjugates for in vivo delivery of polynucleotides
GB2445040B (en) Delivery container
GB0602562D0 (en) Content Delivery
IL194007A0 (en) Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
SG10201406016SA (en) Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
IL198911A0 (en) Drug delivery
EP2088891A4 (de) Verbinder für stift
PL2365064T3 (pl) Warianty fitazy
IL195224A0 (en) Delivery method
EP2056918A4 (de) Katheter für zellenfreisetzung
EP2010162A4 (de) Zusammensetzung zur abgabe eines arzneimittels
EP2023950A4 (de) Krebsbehandlung mit urodilatin
EP1988823A4 (de) Langsame intraventikuläre freisetzung
GB0610866D0 (en) Novel in vivo imaging compounds
EP2035031A4 (de) Insulinzusammensetzung
GB0625783D0 (en) Medicament
GB2444770B (en) Horse boot
GB0615064D0 (en) Medicament
GB2435420B (en) Delivery means
GB0709390D0 (en) Delivery means
GB0618385D0 (en) Instant-action poker
GB0615814D0 (en) Medicament
ZA200708369B (en) Medicament
GB0616952D0 (en) Delivery
GB0610858D0 (en) Novel in vivo imaging compounds

Legal Events

Date Code Title Description
PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

AK Designated contracting states

Kind code of ref document: A2

Designated state(s): AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LT LU LV MC MT NL PL PT RO SE SI SK TR

AX Request for extension of the european patent

Extension state: AL BA HR MK RS

17P Request for examination filed

Effective date: 20090520

RBV Designated contracting states (corrected)

Designated state(s): AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LT LU LV MC MT NL PL PT RO SE SI SK TR

DAX Request for extension of the european patent (deleted)
RAP1 Party data changed (applicant data changed or rights of an application transferred)

Owner name: ARROWHEAD RESEARCH CORPORATION

RAP1 Party data changed (applicant data changed or rights of an application transferred)

Owner name: ARROWHEAD RESEARCH CORPORATION

A4 Supplementary search report drawn up and despatched

Effective date: 20130626

RIC1 Information provided on ipc code assigned before grant

Ipc: A61P 1/16 20060101ALI20130620BHEP

Ipc: A61K 47/48 20060101AFI20130620BHEP

17Q First examination report despatched

Effective date: 20161117

RAP1 Party data changed (applicant data changed or rights of an application transferred)

Owner name: ARROWHEAD PHARMACEUTICALS, INC.

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN

18D Application deemed to be withdrawn

Effective date: 20200211